Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL)

Related Clinical Trial
Tau Protein and SV2a Imaging in Patients With Tau Protein-related Diseases Building Online Community for Parkinson’s Palliative Care Test-retest Study With [18F]PI-2620 in PSP-RS and NDC Evaluation of the Efficacy of a Two-week EMST on Dysphagia in Parkinsonian Patients Optimization of Morphomer-based Alpha-synuclein PET Tracers Evaluation of [18F]APN-1607 as a PET Biomarker A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP) RT001 in Patients With Progressive Supranuclear Palsy (PSP) PROGRESSIVE SUPRANUCLEAR PALSY Complex Eye Movements in Parkinson’s Disease and Related Movement Disorders tDCS and Speech Therapy for Motor Speech Disorders Caused by FTLD Syndromes: a Feasibility Study Application od Machine Learning Method in Validation of Screening Cognitive Test for Parkinsonisms Subcutaneous Apomorphine in the Treatment of Progressive Supranuclear Palsy and Cortico Basal Degeneration (APOPARKA) Remote Monitoring in Progressive Supranuclear Palsy (PSP) Trial of Parkinson’s And Zoledronic Acid Rho Kinase (ROCK) Inhibitor in Tauopathies – 1 UPenn Observational Research Repository on Neurodegenerative Disease Evaluation of Imaging Characteristics of [18F]PI-2620 PET in AD and PSP Patients Using High and Low Specific Activity Systematic Assessment of Laryngopharyngeal Function in Patients With MSA, PD, and 4repeat Tauopathies The MOTIVE-PSP Initiative A Study to Test the Safety and Tolerability of Long-term UCB0107 Administration in Study Participants With Progressive Supranuclear Palsy Efficacy and Safety of Transcranial dIrect Current stiMulation (tDCS) in Progressive Supranuclear Palsy (PSP) (STIM-PSP) Image Characteristic and Longitudinal Follow up of 18F-PMPBB3 (APN-1607) PET for Progressive Supranuclear Palsy Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy Misfolded Proteins in the Skin of People With Parkinson’s Disease and Other Parkinsonism Neurodegenerative Diseases Registry Transcranial Magnetic Stimulation in Progressive Supranuclear Palsy 18F-PM-PBB3 PET Study in Tauopathy Including Alzheimer’s Disease, Other Dementias and Normal Controls Brain Network Activation in Patients With Movement Disorders Neurologic Stem Cell Treatment Study ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) Transcranial Duplex Scanning and Single Photon Emission Computer Tomography (SPECT) in Parkinsonian Syndromes ADDIA Proof-of-Performance Clinical Study Defining Phenotypes of Movement Disorders :Parkinson’s Plus Disorders (PD), Essential Tremor (ET), Cortical Basal Degeneration (CBD), Multiple Systems Atrophy (MSA), Magnetoencephalography. Gait Analysis in Neurological Disease Diagnosing Frontotemporal Lobar Degeneration Evaluation of [18F]MNI-815 as a Potential PET Radioligand for Imaging Tau Protein in the Brain of Patients With Tauopathies Study of the Neural Basis of Analogical Reasoning Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Phase 1 Test-retest Evaluation of [18F]MNI-958 PET Diagnostic and Prognostic Biomarkers in Parkinson Disease Tau Imaging With JNJ067 Research of Biomarkers in Parkinson Disease More Than a Movement Disorder: Applying Palliative Care to Parkinson’s Disease Safety and Efficacy of Droxidopa for Fatigue in Patients With Parkinsonism Identifying Biomarkers of Parkinson’s Disease Using Magnetic Resonance Imaging (MRI) Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL) Davunetide (AL-108) in Predicted Tauopathies – Pilot Study Robot Walking Rehabilitation in Parkinson’s Disease Human CNS Tau Kinetics in Tauopathies 4-Repeat Tauopathy Neuroimaging Initiative – Cycle 2 In-Home Care for Patients With PSP and Related Disorders Facilitating Diagnostics and Prognostics of Parkinsonian Syndromes Using Neuroimaging Study of NBMI Treatment in Patients With Atypical Parkinsons (PSP or MSA) Biomarkers in Parkinsonian Syndromes Evaluation of [18F]MNI-952 as a Potential PET Radioligand for Imaging Tau Protein in the Brain Effects of Coenzyme Q10 in PSP and CBD 2-(1-{6-[(2-[F-18]Fluoroethyl) (Methyl)Amino]-2-naphthyl} Ethylidene) Malononitrile-PET for in Vivo Diagnose of Tauopathy in Unclassified Parkinsonism Oxford Study of Quantification in Parkinsonism A Study to Evaluate the Diagnostic Efficacy of DaTSCAN™ Ioflupane (123I) Injection in Single Photon Emission Computed Tomography (SPECT) for the Diagnosis of Parkinsonian Syndrome (PS) in Chinese Patients Evaluation of [18F]MNI-777 PET as a Marker of Tau Pathology in Subjects With Tauopathies Compared to Healthy Subjects The Neural Basis for Frontotemporal Degeneration Analysis of the Enteric Nervous System Using Colonic Biopsies Evaluation of Tolfenamic Acid in Individuals With PSP at 12-Weeks Chromatic Pupillometry to Assess the Melanopsin-Light Pathway in Progressive Supranuclear Palsy 4 Repeat Tauopathy Neuroimaging Initiative A Study to Assess Tolerability, Safety, Pharmacokinetics and Effect of AZP2006 in Patients With PSP Foot Mechanical Stimulation for Treatment of Gait and Gait Related Disorders in Parkinson’s Disease and Progressive Supranuclear Palsy. Safety Study of TPI-287 to Treat CBS and PSP Phase 0 Evaluation of [18F]MNI-958 as a Potential PET Radioligand for Imaging Tau Protein in the Brain Neuroprotection and Natural History in Parkinson’s Plus Syndromes (NNIPPS) Efficacy Study for Treatment of Dementia in Progressive Supranuclear Palsy A 6 Month, Open-Label, Pilot Futility Clinical Trial of Oral Salsalate for Progressive Supranuclear Palsy Pathophysiology of Gait and Posture in Progressive Supranuclear Palsy A Pilot Clinical Trial of Pyruvate, Creatine, and Niacinamide in Progressive Supranuclear Palsy. Clinical Trial to Evaluate Bone Marrow Stem Cell Therapy for PSP, a Rare Form of Parkinsonism tDCS Plus Physical Therapy for Progressive Supranuclear Palsy Repetitive Transcranial Magnetic Stimulation (TMS) for Progressive Supranuclear Palsy and Corticobasal Degeneration Risk Factors for Progressive Supranuclear Palsy (PSP) Continuously Infused Recombinant-Methionyl Human Glial Cell Line-Derived Neurotrophic Factor (GDNF) to Treat Progressive Supranuclear Palsy Cerebellar rTMS Theta Burst for Postural Instability in Progressive Supranuclear Palsy Neuropsychological Evaluation for Early Diagnosis of PSP Evaluating Cerebrospinal Fluid Biomarkers in Alzheimer’s, Progressive Supranuclear Palsy Subjects, and Controls A Pilot Trial of Lithium in Subjects With Progressive Supranuclear Palsy or Corticobasal Degeneration Extension Study of ABBV-8E12 in Patients With Progressive Supranuclear Palsy (PSP) Who Completed Study C2N-8E12-WW-104 Study About Safety and Efficacy of Coenzyme Q10 in Progressive Supranuclear Palsy Study of the Distractibility Syndrome in Patients With Progressive Supranuclear Palsy Tau Imaging in Subjects With Progressive Supranuclear Palsy, Corticobasal Degeneration and Healthy Volunteers Extension Study of BIIB092 in Participants With Progressive Supranuclear Palsy (PSP) Who Participated in CN002003 PROgressive Supranuclear Palsy CorTico-Basal Syndrome Multiple System Atrophy Longitudinal Study UK The Study to Evaluate the Safety and Efficacy of Spinal Cord Stimulation on Progressive Supranuclear Palsy Study to Evaluate the Safety and Efficacy of Davunetide for the Treatment of Progressive Supranuclear Palsy Young Plasma Transfusions for Progressive Supranuclear Palsy Study of BIIB092 in Participants With Progressive Supranuclear Palsy A Study to Test the Safety and Tolerability of UCB0107 in Study Participants With Progressive Supranuclear Palsy (PSP) Safety, Tolerability, and Pharmacokinetics of C2N-8E12 in Subjects With Progressive Supranuclear Palsy Deep TMS for the Treatment of Patients With Parkinson’s Disease and Progressive Supranuclear Palsy Rehabilitation in Patients With Progressive Supranuclear Palsy A Molecular Anatomic Imaging Analysis of Tau in Progressive Supranuclear Palsy Alpha-lipoic Acid/L-acetyl Carnitine for Progressive Supranuclear Palsy Multiple Ascending Dose Study of Intravenously Administered BMS-986168 (BIIB092) in Patients With Progressive Supranuclear Palsy Effects of Coenzyme Q10 in Progressive Supranuclear Palsy (PSP) Quality of Life of the Patient and the Burden of the Caregiver in Progressive Supranuclear Palsy An Extension Study of ABBV-8E12 in Progressive Supranuclear Palsy (PSP) A Study to Assess Efficacy, Safety, Tolerability, and Pharmacokinetics of ABBV-8E12 in Subjects With Progressive Supranuclear Palsy (PSP). Trial of Valproic Acid in Patients With Progressive Supranuclear Palsy (Depakine) Safety, Tolerability, and Efficacy of Two Different Oral Doses of NP031112 Versus Placebo in the Treatment of Patients With Mild-to-Moderate Progressive Supranuclear Palsy Efficacy, Tolerability and Safety of Azilect in Subjects With Progressive Supranuclear Palsy Postural Instability in Progressive Supranuclear Palsy

Brief Title

Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL)

Official Title

Rare Diseases Clinical Research Network Advancing Research and Treatment for Frontotemporal Lobar Degeneration [ARTFL]: Research Projects 1 & 2

Brief Summary

      Frontotemporal Lobar Degeneration (FTLD) is the neuropathological term for a collection of
      rare neurodegenerative diseases that correspond to four main overlapping clinical syndromes:
      frontotemporal dementia (FTD), primary progressive aphasia (PPA), corticobasal degeneration
      syndrome (CBS) and progressive supranuclear palsy syndrome (PSPS). The goal of this study is
      to build a FTLD clinical research consortium to support the development of FTLD therapies for
      new clinical trials. The consortium, referred to as Advancing Research and Treatment for
      Frontotemporal Lobar Degeneration (ARTFL), will be headquartered at UCSF and will partner
      with six patient advocacy groups to manage the consortium. Participants will be evaluated at
      14 clinical sites throughout North America and a genetics core will genotype all individuals
      for FTLD associated genes.
    

Detailed Description

      Frontotemporal Lobar Degeneration (FTLD) is the neuropathological term for a collection of
      rare neurodegenerative diseases that correspond to four main overlapping clinical syndromes:
      frontotemporal dementia (FTD), primary progressive aphasia (PPA), corticobasal degeneration
      syndrome (CBS) and progressive supranuclear palsy syndrome (PSPS). The goal of this study is
      to build a FTLD clinical research consortium (FTLD CRC) to support the development of FTLD
      therapies for new clinical trials. The FTLD CRC will be headquartered at UCSF and will
      partner with six patient advocacy groups to manage the consortium. Patients will be evaluated
      at 13 clinical sites throughout North America and a genetics core will genotype all
      individuals for FTLD associated genes.

      The study will be divided into 2 projects. The first project will be Preparing for Sporadic
      FTLD Clinical Trials and the second project will be a Longitudinal Assessment of Familial
      FTLD. Self-registration for an online registry will be available for patients and families
      with any FTLD syndrome. Eligible participants for research Projects 1 and 2 FTLD will be
      invited to a CRC site for clinical evaluations. All enrolled participants in both research
      projects will have a site visit consisting of a neurological exam, medical and family
      history, cognitive testing, and a blood draw.

      Participants in Project 1 who have a diagnosis of Progressive Supranuclear Palsy Syndrome
      will have two additional assessments. A lumbar puncture (LP) will be performed for CSF
      collection, and an MRI scan of the brain will be done.

      Participants in Project 2: Longitudinal Assessment of familial FTLD will return for a
      follow-up visit in 12 months; procedures at the follow-up visit will be identical to those at
      baseline. Additionally, asymptomatic participants will undergo MRI scans at both visits.
    


Study Type

Observational


Primary Outcome

Scores of UDS FTLD Module Tests

Secondary Outcome

 Progressive Supranuclear Palsy Rating Scale (PSPRS)

Condition

FTLD


Study Arms / Comparison Groups

 Patients with FTLD or family members
Description:  Participants with FTLD syndrome diagnoses and/or strong family histories of FTLD.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

1489

Start Date

September 2014

Completion Date

September 2020

Primary Completion Date

August 2020

Eligibility Criteria

        1. Inclusion Criteria:Must meet one of the following research diagnostic criteria for a
             Frontotemporal lobar degeneration (FTLD) syndrome: behavioral variant frontotemporal
             dementia (bvFTD), primary progressive aphasia (PPA), semantic variant primary
             progressive aphasia (svPPA), nonfluent variant primary progressive aphasia (nfvPPA),
             frontotemporal dementia with amyotrophic lateral sclerosis (FTD/ALS), amyotrophic
             lateral sclerosis alone, corticobasal syndrome (CBS), progressive supranuclear palsy
             (PSP) or oligosymptomatic PSP (oPSP), or have a strong family history of FTLD
             syndromes.

          2. Between 18 and 85 (inclusive) years of age.

          3. Able to walk (with assistance) at the time of enrollment.

          4. Have a reliable study partner who can provide an independent evaluation of
             functioning.

          5. Speak English or Spanish

          6. Have Mini Mental State Exam (MMSE) scores between 15 - 30 (inclusive).

        Exclusion Criteria:

          1. Known presence of a structural brain lesion (e.g. tumor,cortical infarct) that could
             reasonably explain symptoms in a symptomatic participant without a known f-FTLD
             causing mutation.

          2. Known presence of an Alzheimer's disease causing mutation in PSEN1, PSEN2 or APP; or
             neuropathological evidence for Alzheimer's disease as a cause of syndrome (from brain
             biopsy).

          3. A previous history of Korsakoff encephalopathy, severe alcohol dependence (within 5
             years of onset of dementia), frequent alcohol or other substance intoxication, or
             other neurological disorder (such as multiple sclerosis)

          4. Evidence through history or laboratory testing of B12 deficiency (B12 < 95% of local
             laboratory's normal value), hypothyroidism (TSH >150% of normal), HIV positive,renal
             failure (creatinine > 2), liver failure (ALT or AST > two times normal), respiratory
             failure (requiring oxygen), extra-axial brain tumor (with visible compression of the
             brain parenchyma), large cerebral infarct that could account for clinical syndrome,
             large confluent white matter lesions (grades 3 or 4, [107] significant systemic
             medical illnesses such as deteriorating cardiovascular disease;

          5. Current medication likely to affect CNS functions in the opinion of the site PI: long
             acting benzodiazepines such as diazepam (short-acting benzodiazepines are OK),
             non-SSRI antidepressants (SSRIs or trazodone are OK), no lithium, typical neuroleptics
             as listed in the Manual of Procedures, narcotics (codeine is OK, but hold 24 hours
             before neuropsychological testing), anticonvulsants (outside of therapeutic ranges),
             antihistamines (if taking greater than three times per week; hold 24 hours before
             neuropsychological testing).

          6. In the site investigator's opinion, the participant cannot complete sufficient key
             study procedures, or equivalent assessment of impairment level.

          7. For groups where MRI scans are planned procedures, any contraindication for MRI
             scanning, such as pacemaker or other implanted metals.
      

Gender

All

Ages

18 Years - 85 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Adam L Boxer, MD, PhD, , 

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT02365922

Organization ID

ARTFL8101

Secondary IDs

1U54NS092089-01

Responsible Party

Sponsor

Study Sponsor

University of California, San Francisco

Collaborators

 National Center for Advancing Translational Science (NCATS)

Study Sponsor

Adam L Boxer, MD, PhD, Principal Investigator, Study PI


Verification Date

May 2021